Raptor Pharmaceutical Corporation is pulling the plug on an experimental drug as treatment for a liver disease.
In a statement released on Monday, Sept.14, the biopharmaceutical company said that the drug called RP103 (cysteamine bitartrate) did not meet the main goals in the Phase 2b CyNCh study.
The study, which involved 169 participants between 8 and 17 years old, evaluated the efficacy and safety of RP103 in children diagnosed with nonalcoholic steatohepatitis (NASH), a liver inflammation and damage attributed to the build-up of fat in the liver. The condition can cause scarring of the liver, which could lead to cirrhosis.
For the trial, the participants randomly received either 600 mg per day, 750 mg per day, 900 mg per day of the experimental drug or placebo for a period of 52 weeks. Results showed that the patients who took RP103 did not experience significant improvements in their liver disease.
The drug met its main goal in the first mid-stage study conducted in 2010 but based on the result of the latest clinical trial, the company said that it will no longer pursue the development of the drug for NASH.
Raptor, however, said that it will continue developing the drug for Huntington's disease and mitochondrial diseases. RP103 is also being developed as treatment for the degenerative disease Huntington's and Leigh Syndrome, a neurological disorder.
In a statement, Raptor President Julie Anne Smith expressed disappointment on the outcome of the trial. Nonetheless, she said that the company remains devoted to the development of treatments for people who were diagnosed with rare diseases.
"While these trial results replicated the serological improvements seen in the earlier Phase 2a study, they did not translate into a measurable effect on histology. We're disappointed with this outcome given the paucity of treatment for these children with NASH. While we'll work closely with the NIDDK to understand the full data set, we do not expect to advance this program based on topline results," said Smith.
The results from the trial, which was sponsored by a unit of the National Institute of Health, resulted in the company's share to fall by 34 percent in premarket trading on Monday.
The results of the trial will be submitted for publication and will be presented at the American Association for the Study of Liver Diseases meeting in San Francisco, California this coming November.
Photo: Bill Brooks | Flickr